Section of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden
Sahlgrenska Academy at Gothenburg University, Sweden.
Haematologica. 2017 Oct;102(10):1683-1690. doi: 10.3324/haematol.2017.169862. Epub 2017 Jul 27.
A plastic anemia is a rare life-threatening disease. However, since the introduction of immunosuppressive therapy and allogeneic stem cell transplantation, the outcome has improved considerably, and the 5-year survival is reported to be 70-80% in selected patient cohorts. Yet, contemporary population-based data on incidence and survival are lacking. We performed a national retrospective study to determine the incidence, treatment, and survival of patients with aplastic anemia diagnosed in Sweden from 2000-2011. Patients were included via the National Patient Registry, and diagnosed according to the Camitta criteria. In total, 257 confirmed cases were identified, with an overall incidence of 2.35 (95% CI: 2.06-2.64) cases per million inhabitants per year. Median age was 60 years (range: 2-92), and median follow up was 76 (0-193) months. Primary treatments included immunosuppressive therapy (63%), allogenic stem cell transplantation (10%), or single-agent cyclosporine/no specific therapy (27%). The 5-year survival was 90.7% in patients aged 0-18 years, 90.5% in patients aged 19-39 years, 70.7% in patients aged 40-59 years, and 38.1% in patients aged ≥60 years. Multivariate analysis showed that age (both 40-59 and ≥60 age groups), very severe aplastic anemia and single-agent cyclosporine/no specific therapy were independent risk factors for inferior survival. In conclusion, younger aplastic anemia patients experience a very good long-term survival, while that of patients ≥60 years in particular remains poor. Apparently, the challenge today is to improve the management of older aplastic anemia patients, and prospective studies to address this medical need are warranted.
再生障碍性贫血是一种罕见的危及生命的疾病。然而，自从引入免疫抑制疗法和同种异体干细胞移植以来，其预后已显著改善，在选定的患者队列中，5 年生存率报告为 70-80%。然而，目前缺乏关于发病率和生存率的当代人群数据。我们进行了一项全国性回顾性研究，以确定 2000-2011 年在瑞典诊断为再生障碍性贫血的患者的发病率、治疗方法和生存率。患者通过国家患者登记处纳入研究，并根据卡米塔标准进行诊断。共确定了 257 例确诊病例，总体发病率为每百万居民每年 2.35（95%可信区间：2.06-2.64）例。中位年龄为 60 岁（范围：2-92 岁），中位随访时间为 76（0-193）个月。主要治疗方法包括免疫抑制疗法（63%）、同种异体干细胞移植（10%）或单剂环孢素/无特殊治疗（27%）。0-18 岁患者的 5 年生存率为 90.7%，19-39 岁患者为 90.5%，40-59 岁患者为 70.7%，≥60 岁患者为 38.1%。多变量分析显示，年龄（40-59 岁和≥60 岁组）、极重型再生障碍性贫血和单剂环孢素/无特殊治疗是生存预后不良的独立危险因素。总之，年轻的再生障碍性贫血患者有很好的长期生存，而≥60 岁患者的生存则较差。显然，目前的挑战是改善老年再生障碍性贫血患者的管理，需要进行前瞻性研究来解决这一医疗需求。
